Abstract

Glioblastoma (GBM) is the most common primary brain tumors in adults. Despite aggressive multimodality therapies, GBM unfortunately remains among the most resistant cancers to treatment. In the past, traditional chemotherapy which works by impeding DNA synthesis or cell metabolism has been used to try and slow the progression of GBM with little success. Recently, research has become more focused into the development of targeted therapies in which drugs (small molecules or antibodies) effect specific molecular and genetic alterations in GBM attempting to inhibit and deregulate cell signaling pathways. The Cancer Genome Atlas (TCGA) GBM project has provided an in depth description of the distinct molecular and genetic alterations in GBM stimulating interest in the development of targeted molecular therapies. While the results of targeted therapy studies to date have failed to improve the overall survival of GBM patients, there continues to be enthusiasm in this approach with numerous clinical trials currently underway. Hopefully, knowledge from the previous failed trials will help provide further insight and assist future clinicians in designing new novel targeted treatments to overcome these barriers.

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